PB2670: EQOL-MDS TRIAL: PATIENT-REPORTED OUTCOMES IN PATIENTS WITH LOWER RISK MYELODYSPLASTIC SYNDROMES WITH SEVERE THROMBOCYTOPENIA.
Authors: Esther Oliva, Giuseppe Iannì, Marta Riva, Pasquale Niscola, Valeria Santini, Massimo Breccia, Valentina Gaidano, Antonella Poloni, Andrea Patriarca, Elena Crisà, Isabella Capodanno, Prassede Salutari, Gianluigi Reda, Grazia Sanpaolo, Dario Ferrero, Attilio Guarini, Giovanni Tripepi, Andrea Castelli, Bruno Fattizzo, Germana Beltrami, Monica Bocchia, Alfredo Molteni, Pierre Fenaux, Ulrich Germing, Alessandra Ricco, Giuseppe A. Palumbo, Stefana Impera, Nicola DI Renzo, Francesco Buccisano, Aspasia Stamatoullas, Anna Marina Liberati, Anna Candoni, Ilaria Maria Delfino, Patrizia Cufari, Lorenzo Rizzo, Roberto Latagliata
Journal: HemaSphere (2023)

Topic: 35. Quality of life and palliative care Background: It is well known that quality of life is compromised in myelodysplastic syndromes (MDS). The impact of severe thrombocytopenia and its treatment in such patients has not yet been reported. EQOL-MDS is an international, multicenter, randomized, single-blind, placebo-controlled trial to evaluate safety and efficacy of eltrombopag for the treatment of severe thrombocytopenia of lower risk MDS. Primary endpoints (platelet response in the first 24 weeks, safety, duration of platelet response) have been reached and previously published, favouring eltrombopag with a response rate of 42% versus 11% for placebo. Aims: The aim is to evaluate, as a secondary endpoint, the changes in patient-reported outcome measures (PROMs) at 24 weeks. Methods: Main inclusion criteria are age ≥ 18 years with morphological diagnosis of low or intermediate-1 international prognostic scoring system MDS with stable platelet count <30 Gi/L. 169 participants (104 males; median age 72 [IQR 65 -79] years) were randomly assigned (2:1) to receive eltrombopag or matching placebo at an initial dose of 50 mg once daily, titrated in 50 mg increments every 2 weeks up to a maximum of 300 mg to achieve and maintain a complete platelet response, defined as a platelet count ≥100 Gi/L without bleeding. PROMs included EORTC QLQ-C30 and the MDS-specific instrument, QOL-E, assessed at baseline and at 24 weeks. Results: For those achieving a platelet response, there were proportionally more cases with stability or clinically meaningful and significant improvement in PROMS with respect to non-responders who had a significantly higher proportion of worsening (Table). Summary/Conclusion: This is the first assessment of PROMs in patients with lower risk MDS receiving treatment for severe thrombocytopenia in a randomized trial. Changes in platelet counts were associated with clinically meaningful improvements in quality of life and symptoms which confers further value to treatment with eltrombopag beyond increasing platelet counts. Table - QOL-E Domain changes at 24 weeks Responders (%) Non-responders (%) p-value Functional Worsened 15.0 42.4 0.023 Stable 50.0 39.4 Improved 35.0 18.2 Physical Worsened 43.5 45.7 0.732 Stable 21.7 25.7 Improved 34.8 28.6 Social Worsened 16.0 33.3 0.178 Stable 60.0 48.5 Improved 24.0 18.2 Fatigue Worsened 8.7 45.7 < 0.0001 Stable 47.8 37.1 Improved 43.5 17.1 MDS-Specific Worsened 6.3 50.0 0.004 Stable 68.8 34.6 Improved 24.9 15.4 General Worsened 9.1 40.0 0.012 Stable 54.5 50.0 Improved 36.4 10.0 All Worsened 11.1 43.8 0.018 Stable 44.5 43.8 Improved 44.4 12.4 Treatment-outcome index Worsened 8.3 60.9 0.007 Stable 58.3 17.4 Improved 33.4 21.7 EORTC QLQ-C30 Domain changes at 24 weeks Responders (%) Non-responders (%) p-value Global Health Status Worsened 11.1 37.8 0.010 Stable 40.7 35.2 Improved 48.2 27.0 Physical Worsened 11.5 41.7 0.028 Stable 57.7 36.1 Improved 30.8 22.2 Role function Worsened 8.0 36.1 0.260 Stable 60.0 27.8 Improved 32.0 36.1 Emotional Worsened 14.8 32.4 0.136 Stable 59.3 48.6 Improved 25.9 19.0 Cognitive Worsened 33.3 27.0 0.948 Stable 37.0 48.7 Improved 29.7 24.3 Social Worsened 29.6 45.9 0.594 Stable 59.3 32.5 Improved 11.1 21.6 Fatigue Worsened 19.2 25.7 0.832 Stable 57.7 48.6 Improved 23.1 25.7 Keywords: Myelodysplastic syndrome, Clinical trial, Thrombocytopenia, Quality of life